In a First, Gene Therapy Halts a Fatal Brain Disease
For the first time, doctors have used gene therapy to stave off a fatal degenerative brain disease, an achievement that some experts had thought impossible.
The key to making the therapy work? One of the medicine’s greatest villains: HIV.
The patients were children who had inherited a mutated gene causing a rare disorder, adrenoleukodystrophy, or ALD. Nerve cells in the brain die, and in a few short years, children lose the ability to walk or talk. More...